New gene therapy aims to halt devastating nerve disease in children
Disease control
Ongoing
This study is testing a one-time gene therapy called FBX-101 for children with Krabbe disease, a rare and severe genetic disorder that damages the nervous system. The therapy is given as an infusion to children who have already received a stem cell transplant, which is the curren…
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC